Splice-region antisense composition and method
First Claim
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1. A pharmaceutical composition comprising an oligomer of 8 to 40 morpholino subunits connected by phosphorous-containing intersubunit linkages, wherein the 5′
- terminal morpholino subunit comprises a group of the formula;
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Abstract
Antisense compositions targeted against an mRNA sequence coding for a selected protein, at a region having its 5′ end from 1 to about 25 base pairs downstream of a normal splice acceptor junction in the preprocessed mRNA, are disclosed. The antisense compound is RNase-inactive, and is preferably a phosphorodiamidate-linked morpholino oligonucleotide. Such targeting is effective to inhibit natural mRNA splice processing, produce splice variant mRNAs, and inhibit normal expression of the protein.
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13 Claims
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1. A pharmaceutical composition comprising an oligomer of 8 to 40 morpholino subunits connected by phosphorous-containing intersubunit linkages, wherein the 5′
- terminal morpholino subunit comprises a group of the formula;
- View Dependent Claims (2, 3, 4, 5, 6, 7, 8, 9, 10, 11)
- terminal morpholino subunit comprises a group of the formula;
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12. A pharmaceutical composition comprising a compound of formula:
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13. A pharmaceutical composition comprising a compound of formula:
Specification